"Vertex continues to make significant progress with the key growth
drivers for our business - increasing the number of people being treated
with ORKAMBI and KALYDECO, expanding the number of people eligible for
these medicines through label-expansions and developing new medicines to
treat potentially all people with CF in the future," said Jeffrey
Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of
Vertex. "Our progress toward treating more people with CF was marked by
several important milestones in recent weeks, including the approval of
ORKAMBI for children ages six to eleven in the U.S. and today's
announcement regarding the advancement of our pipeline of
next-generation correctors. Importantly, we're also continuing to
generate important additional data about the long-term benefits of
treating the underlying cause of CF with both ORKAMBI and KALYDECO."

Vertex today reviewed recent progress from across its CF program:

ORKAMBI

FDA approval of ORKAMBI for the treatment of children ages 6 to 11:
On September 28, 2016 the U.S. Food and Drug Administration (FDA)
approved ORKAMBI for the treatment of children ages 6 through 11 who
have two copies of the F508del mutation. There are approximately 2,400
children ages 6 through 11 who have two copies of the F508del mutation
in the U.S.

Data from Phase 3 efficacy study to support approval in children
ages 6 to 11 in Europe expected by year-end: Vertex completed
enrollment in a six-month Phase 3 efficacy study evaluating ORKAMBI in
children ages 6 through 11 who have two copies of the F508del mutation
and expects data from this study by the end of 2016. The primary
endpoint of the study is the absolute change in lung clearance index.
Pending data from the study, Vertex plans to submit a Marketing
Authorization Application variation in the European Union in the first
half of 2017. In Europe, there are approximately 3,400 children ages 6
through 11 who have two copies of the F508del mutation.

Tezacaftor (VX-661) in Combination with Ivacaftor

Enrollment complete in two Phase 3 studies of tezacaftor (VX-661);
data expected in first half of 2017: Vertex has now completed
enrollment in two of three ongoing Phase 3 studies of the
investigational combination of tezacaftor and ivacaftor. Enrollment is
complete in the Phase 3 study in people ages 12 and older who have two
copies of the F508del mutation and also in the Phase 3 study in people
ages 12 and older who have one F508del mutation and one residual
function mutation. Data from both studies are expected in the first half
of 2017. The Phase 3 study of tezacaftor in combination with ivacaftor
in people with one F508del mutation and one gating mutation is expected
to complete enrollment in early 2017. Vertex plans to submit a New Drug
Application (NDA) to the FDA for tezacaftor in combination with
ivacaftor in the second half of 2017, pending data from the Phase 3
program.

Next-Generation Correctors

Planned initiation of Phase 2 studies in CF patients: In a
separate press release issued today, Vertex announced that it plans to
initiate two Phase 2 studies to evaluate the next-generation correctors
VX-440 and VX-152 in triple combination regimens with tezacaftor
(VX-661) and ivacaftor in people with cystic fibrosis (CF). Both studies
are expected to start by the end of 2016. Additional details on the
design of these studies were provided today in a separate press release.

Additional next-generation correctors moving into clinical
development: Vertex also today announced that it plans to begin
a Phase 1 study of VX-659, the company's third next-generation
corrector, by the end of 2016 and to advance a fourth next-generation
corrector into clinical development in 2017. Additional details were
provided today in a separate press release.

Third Quarter 2016 Financial Highlights

Revenues:

Net product revenues from ORKAMBI were $234.0 million compared to
$130.8 million for the third quarter of 2015. ORKAMBI was launched in
the U.S. in July 2015.

Net product revenues from KALYDECO were $175.6 million, compared to
$165.9 million for the third quarter of 2015.

Expenses:

GAAP operating expenses were $435.5 million compared to $379.8 million
for the third quarter of 2015. Non-GAAP operating expenses (combined
non-GAAP R&D and SG&A) were $298.0 million compared to $277.7 million
for the third quarter of 2015. The increases were primarily driven by
increased costs related to the progression of our CF pipeline and to
increased investment in global commercial support for the launch of
ORKAMBI.

GAAP R&D expenses were $275.4 million compared to $246.3 million for
the third quarter of 2015. Non-GAAP R&D expenses were $214.0 million
compared to $201.6 million for the third quarter of 2015. The
increases were primarily driven by increased investment to progress
our portfolio of CF medicines.

GAAP SG&A expenses were $106.1 million compared to $99.8 million for
the third quarter of 2015. Non-GAAP SG&A expenses were $84.0 million
compared to $76.1 million for the third quarter of 2015. The increases
were primarily driven by increased investment to support the global
launch of ORKAMBI.

Net Income (Loss) Attributable to Vertex:

GAAP net loss was $(41.8) million, or $(0.17) per diluted share,
compared to GAAP net loss of $(95.1) million, or $(0.39) per diluted
share, for the third quarter of 2015. Non-GAAP net income was $40.1
million, or $0.16 per diluted share, compared to a non-GAAP net loss
of $(31.9) million, or $(0.13) per diluted share, for the third
quarter of 2015.

Cash Position:

As of September 30, 2016, Vertex had $1.13 billion in cash, cash
equivalents and marketable securities compared to $1.04 billion in
cash, cash equivalents and marketable securities as of December 31,
2015.

As of September 30, 2016, Vertex had $300 million outstanding from a
credit agreement, which was refinanced on October 13, 2016 to lower
the company's interest expense. The $300 million outstanding under the
new credit agreement matures in the fourth quarter of 2021.

2016 Financial Guidance:

Vertex today reiterated its 2016 revenue guidance for ORKAMBI and
KALYDECO. The company also reiterated guidance for its 2016 combined
non-GAAP R&D and SG&A expenses. The guidance is summarized below:

ORKAMBI: The company continues to expect total 2016 product
revenues for ORKAMBI of $950 to $990 million.

KALYDECO: The company continues to expect total 2016 product
revenues for KALYDECO of $685 to $705 million. 2016 guidance for
KALYDECO currently excludes any revenues related to the potential
approval of KALYDECO for people in the U.S. who have residual function
mutations.

Operating Expenses (Combined Non-GAAP R&D and SG&A Expenses): Vertex
continues to expect that its combined non-GAAP R&D and SG&A expenses
in 2016 will be in the range of $1.18 to $1.23 billion. Vertex's
expected non-GAAP R&D and SG&A expenses exclude stock-based
compensation expense and certain other expenses.

Non-GAAP Financial Measures

In this press release, Vertex's financial results and financial guidance
are provided in accordance with accounting principles generally accepted
in the United States (GAAP) and using certain non-GAAP financial
measures. In particular, non-GAAP financial results and guidance exclude
stock-based compensation expense, revenues and expenses related to
consolidated variable interest entities, costs and credits related to
the relocation of the company's corporate headquarters and hepatitis
C-related revenues and costs and other adjustments. These results are
provided as a complement to results provided in accordance with GAAP
because management believes these non-GAAP financial measures help
indicate underlying trends in the company's business, are important in
comparing current results with prior period results and provide
additional information regarding the company's financial position.
Management also uses these non-GAAP financial measures to establish
budgets and operational goals that are communicated internally and
externally and to manage the company's business and to evaluate its
performance. The company adjusts, where appropriate, for both revenues
and expenses in order to reflect the company's operations. The company
provides guidance regarding product revenues in accordance with GAAP and
provides guidance regarding combined non-GAAP research and development
and sales, general, and administrative expenses. The company does not
provide guidance regarding GAAP research and development and sales,
general, and administrative expenses because of the difficulty of
estimating stock-based compensation expenses, and predicting whether or
not there will be additional expense items for which adjustments are
appropriate. A reconciliation of the GAAP financial results to non-GAAP
financial results is included in the attached financial information.

Note 1 : Cost of product revenues in the nine months ended
September 30, 2016 includes the second and final $13.9 million
commercial milestone that was earned by CFFT in the first quarter of
2016 related to sales of ORKAMBI.

Note 2: The company excludes restructuring expense (income) from
its non-GAAP income (loss) attributable to Vertex. In the three and nine
months ended September 30, 2016 and 2015, "Real estate restructuring
costs and income" consisted of restructuring charges related primarily
to the company's relocation from Cambridge to Boston, Massachusetts.

Note 3: In the three and nine months ended September 30, 2016 and
2015, "HCV related revenues and costs" included net product revenues
from Incivek, royalty revenues from Incivo, HCV collaborative revenues
and operating costs and expenses related to HCV. The Company withdrew
Incivek from the market in the United States in 2014.

Note 4: In the three months ended September 30, 2016, "Other
adjustments" was primarily attributable to payments for collaborations.
In the nine months ended September 30, 2016, "Other adjustments" was
primarily attributable to a $58.5 million increase in the fair value of
contingent milestone payments and royalties payable by Vertex to Parion
due to the Phase 2 study meeting its primary safety endpoint and
payments for collaborations and the acquisition of certain early stage
assets.

Note 5: The company consolidates the financial statements of two
of its collaborators as variable interest entities ("VIEs") as of
September 30, 2016 and December 31, 2015. These VIEs are consolidated
because Vertex has licensed the rights to develop the company's
collaborators' most significant intellectual property assets. The
company's interest and obligations with respect to these VIEs' assets
and liabilities are limited to those accorded to the company in its
collaboration agreements with these collaborators. Restricted cash and
cash equivalents (VIE) reflects the VIEs' cash and cash equivalents,
which Vertex does not have any interest in and which will not be used to
fund the collaboration. Each reporting period Vertex estimates the fair
value of the contingent milestone payments and royalties payable by
Vertex to these collaborators. Any increase in the fair value of these
contingent milestone and royalty payments results in a decrease in net
income attributable to Vertex (or an increase in net loss attributable
to Vertex) on a dollar-for-dollar basis. The fair value of contingent
milestone and royalty payments is evaluated each quarter and any change
in the fair value is reflected in the company's statement of operations.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO®
(ivacaftor)

KALYDECO (ivacaftor) is a prescription medicine used for the treatment
of cystic fibrosis (CF) in patients age 2 years and older who have one
of the following mutations in their CF gene: G551D, G1244E,
G1349D, G178R, G551S, S1251N, S1255P, S549N,
S549R, or R117H. KALYDECO is not for use in people with CF
due to other mutations in the CF gene. KALYDECO is not effective in
patients with CF with two copies of the F508del mutation (F508del/F508del)
in the CF gene. It is not known if KALYDECO is safe and effective in
children under 2 years of age.

Patients should not take KALYDECO if they are taking certain
medicines or herbal supplements such as: the antibiotics rifampin or
rifabutin; seizure medications such as phenobarbital, carbamazepine, orphenytoin; or St. John's wort.

Before taking KALYDECO, patients should tell their doctor if they:
have liver or kidney problems; drink grapefruit juice, or eat grapefruit
or Seville oranges; are pregnant or plan to become pregnant because it
is not known if KALYDECO will harm an unborn baby; and are breastfeeding
or planning to breastfeed because is not known if KALYDECO passes into
breast milk.

KALYDECO may affect the way other medicines work, and other medicines
may affect how KALYDECO works. Therefore the dose of KALYDECO may
need to be adjusted when taken with certain medications. Patients should
especially tell their doctor if they take antifungal medications such as
ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole;
or antibiotics such as telithromycin, clarithromycin, or erythromycin.

KALYDECO can cause dizziness in some people who take it. Patients should
not drive a car, use machinery, or do anything that needs them to be
alert until they know how KALYDECO affects them. Patients should avoid
food containing grapefruit or Seville oranges while taking KALYDECO.

KALYDECO can cause serious side effects including:

High liver enzymes in the blood have been reported in patients
receiving KALYDECO. The patient's doctor will do blood tests to
check their liver before starting KALYDECO, every 3 months during the
first year of taking KALYDECO, and every year while taking KALYDECO. For
patients who have had high liver enzymes in the past, the doctor may do
blood tests to check the liver more often. Patients should call their
doctor right away if they have any of the following symptoms of liver
problems: pain or discomfort in the upper right stomach (abdominal)
area; yellowing of their skin or the white part of their eyes; loss of
appetite; nausea or vomiting; or dark, amber-colored urine.

Abnormality of the eye lens (cataract) has been noted in some children
and adolescents receiving KALYDECO. The patient's doctor should perform
eye examinations prior to and during treatment with KALYDECO to look for
cataracts. The most common side effects include headache; upper
respiratory tract infection (common cold), which includes sore throat,
nasal or sinus congestion, and runny nose; stomach (abdominal) pain;
diarrhea; rash; nausea; and dizziness.

These are not all the possible side effects of KALYDECO.

Please click here
to see the full Prescribing Information for KALYDECO (ivacaftor).

INDICATION AND IMPORTANT SAFETY INFORMATION FOR ORKAMBI® (lumacaftor/ivacaftor)
TABLETS

ORKAMBI is a prescription medicine used for the treatment of cystic
fibrosis (CF) in patients age 6 years and older who have two copies of
the F508del mutation (F508del/F508del) in their CFTR gene.
ORKAMBI should only be used in these patients. It is not known if
ORKAMBI is safe and effective in children under 6 years of age.

Patients should not take ORKAMBI if they are taking certain medicines
or herbal supplements, such as: the antibiotics rifampin or
rifabutin; the seizure medicines phenobarbital, carbamazepine, or
phenytoin; the sedatives/anti-anxiety medicines triazolam or midazolam;
the immunosuppressant medicines everolimus, sirolimus, or tacrolimus; or
St. John's wort.

Before taking ORKAMBI, patients should tell their doctor if they: have
or have had liver problems; have kidney problems; have had an organ
transplant; are using birth control (hormonal contraceptives, including
oral, injectable, transdermal or implantable forms). Hormonal
contraceptives should not be used as a method of birth control when
taking ORKAMBI. Patients should tell their doctor if they are pregnant
or plan to become pregnant (it is unknown if ORKAMBI will harm the
unborn baby) or if they are breastfeeding or planning to breastfeed (it
is unknown if ORKAMBI passes into breast milk).

ORKAMBI may affect the way other medicines work and other medicines may
affect how ORKAMBI works. Therefore, the dose of ORKAMBI or other
medicines may need to be adjusted when taken together. Patients should
especially tell their doctor if they take: antifungal medicines such as
ketoconazole, itraconazole, posaconazole, or voriconazole; or
antibiotics such as telithromycin, clarithromycin, or erythromycin.

When taking ORKAMBI, patients should tell their doctor if they
stop ORKAMBI for more than 1 week as the doctor may need to change the
dose of ORKAMBI or other medicines the patient is taking. It is unknown
if ORKAMBI causes dizziness. Patients should not drive a car, use
machinery, or do anything requiring alertness until the patient knows
how ORKAMBI affects them.

ORKAMBI can cause serious side effects including:

High liver enzymes in the blood, which can be a sign of liver injury,
have been reported in patients receiving ORKAMBI. The patient's
doctor will do blood tests to check their liver before they start
ORKAMBI, every three months during the first year of taking ORKAMBI, and
annually thereafter. The patient should call the doctor right away if
they have any of the following symptoms of liver problems: pain or
discomfort in the upper right stomach (abdominal) area; yellowing of the
skin or the white part of the eyes; loss of appetite; nausea or
vomiting; dark, amber-colored urine; or confusion.

Respiratory events such as shortness of breath or chest tightness
were observed in patients when starting ORKAMBI. If a patient has
poor lung function, their doctor may monitor them more closely when
starting ORKAMBI.

An increase in blood pressure has been seen in some patients treated
with ORKAMBI. The patient's doctor should monitor their blood
pressure during treatment with ORKAMBI.

Abnormality of the eye lens (cataract) has been noted in some
children and adolescents receiving ORKAMBI and ivacaftor, a component of
ORKAMBI. For children and adolescents, the patient's doctor should
perform eye examinations prior to and during treatment with ORKAMBI to
look for cataracts.

Please click here
to see the full Prescribing Information for ORKAMBI.

About Vertex

Vertex is a global biotechnology company that aims to discover, develop
and commercialize innovative medicines so people with serious diseases
can lead better lives. In addition to our clinical development programs
focused on cystic fibrosis, Vertex has more than a dozen ongoing
research programs aimed at other serious and life-threatening diseases.

Founded in 1989 in Cambridge, Mass., Vertex today has research and
development sites and commercial offices in the United States, Europe,
Canada and Australia. For six years in a row, Science magazine
has named Vertex one of its Top Employers in the life sciences. For
additional information and the latest updates from the company, please
visit www.vrtx.com.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including, without
limitation, Dr. Leiden's statements in the second paragraph of the press
release, the information provided in the section captioned "2016
Financial Guidance" and statements regarding (i) the expected timing and
clinical trial designs for ongoing and planned clinical studies of
ORKAMBI, tezacaftor (VX-661), and the company's next-generation
correctors, including VX-659, and (ii) the timing of regulatory
applications, including NDA and MAAs. While Vertex believes the
forward-looking statements contained in this press release are accurate,
these forward-looking statements represent the company's beliefs only as
of the date of this press release and there are a number of factors that
could cause actual events or results to differ materially from those
indicated by such forward-looking statements. Those risks and
uncertainties include, among other things, that the company's
expectations regarding its 2016 revenues and expenses may be incorrect
(including because one or more of the company's assumptions underlying
its expectations may not be realized), that data from the company's
development programs may not support registration or further development
of its compounds due to safety, efficacy or other reasons, and other
risks listed under Risk Factors in Vertex's annual report and quarterly
reports filed with the Securities and Exchange Commission and available
through the company's website at www.vrtx.com.
Vertex disclaims any obligation to update the information contained in
this press release as new information becomes available.

Conference Call and Webcast

The company will host a conference call and webcast today at 4:30 p.m.
ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720)
545-0001 (International). The conference call will be webcast live and a
link to the webcast can be accessed through Vertex's website at www.vrtx.com
in the "Investors" section under "Events and Presentations." To ensure a
timely connection, it is recommended that users register at least 15
minutes prior to the scheduled webcast. An archived webcast will be
available on the company's website.